Savara Presented Data at the European Respiratory Society (ERS) International Congress 2022

Rhea-AI Summary

Savara Inc. (NASDAQ: SVRA) presented three posters at the ERS International Congress 2022 in Barcelona, focusing on autoimmune pulmonary alveolar proteinosis (aPAP). Key findings include a post hoc analysis from the IMPALA trial showing improved gas exchange and health status in patients treated with inhaled molgramostim compared to placebo. Safety data from the IMPALA-X study indicated no new safety signals. The company highlighted the rationale for using DLco as a primary endpoint in the ongoing IMPALA-2 trial, aiming to demonstrate the efficacy of molgramostim in aPAP.

Positive

• Significant improvements in gas exchange and health status for molgramostim-treated patients in the IMPALA trial.
• No new safety signals reported in the IMPALA-X study after 93.4 patient-years of exposure.
• Ongoing IMPALA-2 trial aims to further validate molgramostim's efficacy in treating aPAP.

Negative

• The IMPALA-X study was discontinued early due to ineffective dosing regimen based on prior trial results.

AUSTIN, Texas--(BUSINESS WIRE)-- Savara Inc. (Nasdaq: SVRA), a clinical stage biopharmaceutical company focused on rare respiratory diseases, presented three posters at the ERS International Congress 2022 that took place September 4-6^th in Barcelona, Spain.

Below are summaries of each poster presented:

Abstract #2170: “IMPALA: Efficacy Measures in Patients with Autoimmune Pulmonary Alveolar Proteinosis (aPAP) Who Required Whole Lung Lavage” presented by Y. Inoue, M.D., Ph.D.

• Presented data from a post hoc analysis of the IMPALA clinical trial evaluating the benefit of continuous daily administration of inhaled molgramostim 300 μg vs placebo among patients with aPAP who required whole lung lavage (WLL) during the double-blind treatment period. A total of 10 patients underwent WLL in the treatment period (molgramostim: n=4, placebo: n=6)
• Data demonstrated that patients treated with molgramostim after undergoing WLL had greater improvements in endpoint measures of gas exchange, including alveolar-arterial oxygen difference (A-aDO₂) and diffusing capacity of the lungs for carbon monoxide (DLco) corrected for hemoglobin levels. Additionally, patients reported improved health status, as measured by St. George’s Respiratory Questionnaire-Total and -Activity, when compared with placebo-treated patients .
• Click here to view the poster

Abstract #1154: “Safety and Tolerability of Inhaled Molgramostim in Autoimmune Pulmonary Alveolar Proteinosis (aPAP)” presented by F. Bonella, M.D., Ph.D.

• Included safety data from an open-label, non-controlled extension study of IMPALA (IMPALA-X) in which 60 patients received treatment with inhaled molgramostim 300 μg/day administered intermittently (1 week on and 1 week off). This study was discontinued early as results from IMPALA did not support this dosing regimen.
• In IMPALA-X, no new safety signals were observed and the most common treatment-related adverse events after 93.4 patient-years of exposure were mild-to-moderate cough, nasopharyngitis, and respiratory tract infection.
• Click here to view the poster

Abstract #3136: “IMPALA-2: Choice of DLco as a Primary Endpoint in Autoimmune Pulmonary Alveolar Proteinosis (aPAP)” presented by B. Trapnell, M.D.

• Described rationale for using DLco as a primary endpoint in the IMPALA-2 clinical trial, including:
  • In the first IMPALA trial, DLco showed robust improvements in patients with aPAP who were treated with continuous daily molgramostim 300 μg compared to those treated with placebo
  • DLco is a standardized, widely used lung function test with direct relevance to the pathophysiology of aPAP, and is predictive for the major clinical event of WLL
• IMPALA-2 is an ongoing pivotal Phase 3, randomized, double-blind, placebo-controlled global clinical trial evaluating once daily molgramostim 300 μg in patients with aPAP
• Click here to view the poster

The full content of these posters is available in the Congress’ online program and the Articles and Publications page of the Savara corporate website. Additionally, the posters are scheduled to be published in a supplement of the European Respiratory Journal (ERJ) by the end of November 2022. For more details about the ERS International Congress please visit https://www.ersnet.org/congress-and-events/congress/.

About aPAP

Autoimmune pulmonary alveolar proteinosis (aPAP) is a rare lung disease that belongs to a family of distinct rare lung diseases collectively known as pulmonary alveolar proteinosis (PAP). aPAP represents about 90% of all patients with PAP. While aPAP can affect people of any age, race or sex, onset occurs most frequently in people between the ages of 30 and 40. PAP is characterized by the build-up of surfactant in the alveoli, or air sacs, of the lungs. The surfactant consists of proteins and lipids and is an important physiological substance that lines the inside of the alveoli to prevent the lungs from collapsing. The root cause of aPAP is an autoimmune response against GM-CSF, a naturally occurring protein in the body. Pulmonary macrophages need to be stimulated by GM-CSF to function properly, but in aPAP, GM-CSF is neutralized by antibodies against GM-CSF, rendering the macrophages unable to perform their tasks, including the clearance of surfactant from the alveoli. In aPAP, the feeling of having trouble breathing is the most common symptom. People with aPAP can also experience chronic cough, fatigue, sputum production, reduced ability to exercise and episodes of fever due to underlying pulmonary infections. There are currently no approved pharmaceutical treatment options for aPAP.

About Savara

Savara is a clinical stage biopharmaceutical company focused on rare respiratory diseases. Our lead program, molgramostim nebulizer solution, is an inhaled granulocyte-macrophage colony-stimulating factor (GM-CSF) in Phase 3 development for autoimmune pulmonary alveolar proteinosis (aPAP). Molgramostim is delivered via an investigational eFlow® Nebulizer System (PARI Pharma GmbH). Our management team has significant experience in rare respiratory diseases and pulmonary medicine, identifying unmet needs, and effectively advancing product candidates to approval and commercialization. More information can be found at www.savarapharma.com. (Twitter: @SavaraPharma, LinkedIn: www.linkedin.com/company/savara-pharmaceuticals/).

View source version on businesswire.com: https://www.businesswire.com/news/home/20220906005655/en/

Savara Inc. IR & PR

Anne Erickson

Senior Vice President, Chief of Staff

anne.erickson@savarapharma.com

(512) 851-1366

Source: Savara Inc.

FAQ

What are the key findings from Savara's presentation at the ERS International Congress 2022?

Savara presented three posters showing improved gas exchange and health status in aPAP patients treated with molgramostim compared to placebo.

What is the status of the IMPALA-2 clinical trial for Savara?

The IMPALA-2 trial is ongoing, evaluating the efficacy of molgramostim in patients with autoimmune pulmonary alveolar proteinosis.

Were there any safety concerns reported during the clinical trials?

No new safety signals were observed in the IMPALA-X study after extensive patient exposure.

What does aPAP stand for and why is it significant?

aPAP stands for Autoimmune Pulmonary Alveolar Proteinosis, a rare lung disease with currently no approved pharmaceutical treatments.